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11:40 am

Tue December 24, 2013

Drugmaker Names Pill After CEO Who Sought Daughter's Cure

Martine Rothblatt, CEO of United Therapeutics, is the namesake of the company's latest drug, Orenitram.

Ron LevineGetty Images

It's not every day that the Food and Drug Administration approves a drug three months ahead of schedule. Or approves a pill that could take the place of injections. Or gives the OK to a medicine named for the CEO who started a company to help her sick daughter.

But all those things just happened.

United Therapeutics' Orenitram got the FDA's approval as a treatment for pulmonary arterial hypertension. The life-threatening illness is caused by constriction of the arteries that carry blood from the heart to the lungs. It's marked by fatigue, shortness of breath, chest pain and heart failure.

Drugs that relax the arteries can help. But a core treatment used to be available only by injection.

"When I started the company 17 years ago, the only option for treating pulmonary hypertension was intravenous prostacyclin," Martine Rothblatt, CEO of United Therapeutics told investors in a celebratory conference call Monday. "My young daughter looked at me and said, 'Can't there be a pill?' "

Now there is: Orenitram. The name comes from "Martine Ro," as in Martine Rothblatt, backward.

"What it has resulted in is something that as the parent of a lovely young lady with pulmonary hypertension, I cannot even put into words, the emotional meaning that it has to have a pill form of prostacyclin," Rothblatt said.

A little later in the call, she tried. "When something takes a long time to bake, be it a turkey or a cake, the bad thing is that you're hungry and you're starving for a long time," Rothblatt said. "But the good thing is you usually have an awesome meal at the end. It's a very, very similar situation with Orenitram. This baby has taken a long time to bake — no doubt about it."

There are many drugs available for pulmonary hypertension, including pills containing the same active ingredients as Viagra and Cialis. But Orenitram marks a first in the U.S. — a prostacyclin that can be taken by mouth, the company said.

Orenitram won't be available for sale for about six months, the company said, and its exact price hasn't been determined either. But it will be very expensive, about $150,000 a year, according to an estimate by Dr. Mark Schoenebaum, an industry analyst with ISI.

Insurers generally pay for pulmonary hypertension drugs, though there were questions from analysts about whether health plans would pay for Orenitram if it's used in combination with other drugs.

The FDA approved Orenitram on the basis of a study that gauged its safety and effectiveness as a standalone treatment. The drug helped people walk a little farther in a timed walking test. The most common side effects seen in the study were headache, nausea and diarrhea.

Rothblatt acknowledged that the cost of the drugs is high but manageable, because pulmonary hypertension remains pretty rare, though the exact prevalence is hard to pin down.

There's far more to the Orenitram story. Martine Rothblatt used to be Martin, a founder of Sirius Satellite Radio, before undergoing gender reassignment surgery and becoming a drug company executive. For more on Rothblatt and United Therapeutics, read the 2010 profile by Forbes' Matthew Herper.

Finally, you check out Rothblatt honoring daughter Jenesis at the 15th anniversary of the founding of the company in the video below.